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August 2 - August 8, 2018
As long as the genetic code for a particular trait is known, scientists can use CRISPR to insert, edit, or delete the associated gene in virtually any living plant’s or animal’s genome.
Researchers have corrected the DNA mistakes that cause Duchenne muscular dystrophy by snipping out only the damaged region of the mutated gene, leaving the rest intact. In the case of hemophilia A, researchers have harnessed CRISPR to precisely rearrange over half a million letters of DNA that are inverted in the genomes of affected patients. CRISPR might even be used to treat HIV/AIDS, either by cutting the virus’s DNA out of a patient’s infected cells or by editing the patient’s DNA so that the cells avoid infection altogether.
A global discussion about gene editing has already begun; it’s a historic debate about nothing less than the future of our world. The wave is coming. Let’s paddle out and ride it together.
This overall flow of genetic information—from DNA to RNA to protein—is known as the central dogma of molecular biology, and it is the language used to communicate and express life.
The human genome also includes a separate mini-chromosome—just sixteen thousand letters of DNA—located in mitochondria, the energy-producing batteries of the cell. Unlike the genetic code found in other chromosomes, mitochondrial DNA is inherited exclusively from the mother.
while genome sequencing represents a huge development in the study of genetic disease, it is ultimately a diagnostic tool, not a form of treatment. It has allowed us to see how genetic diseases are written in the language of DNA, but it leaves us powerless to change that language. After all, it’s one thing to learn how to read; it’s quite another to learn how to write.
the idea of treating genetic disease by rewriting the mutated genetic code seemed impossible. Repairing a defective gene would be like finding a needle in a haystack and then removing that needle without disturbing a single strand of hay in the process.
Inspired in part by the uncanny ability of viruses to splice new genetic information into the DNA of bacterial cells, the pioneers of this early gene therapy realized they could use viruses to deliver therapeutic genes to humans.
A full 8 percent of the human genome—over 250 million letters of DNA—is a remnant of ancient retroviruses that infected ancestors of our species millennia ago.
Would it be a blessing or a curse to suddenly be rid of the winged pests that have inhabited the earth for more than one hundred million years? Somewhat incredibly, scientists don’t seem overly concerned about a world without mosquitoes. As one entomologist put it, “If we eradicated them tomorrow, the ecosystems where they are active will hiccup and then get on with life.” If he’s right and we could have a world free from the ravages of mosquito-borne illness, can we justify not taking the risk?
Vice President Biden’s invitation was perhaps the most resounding affirmation of this technology’s significance for the field of public health. Just as powerful as the implications for CRISPR research was the reason behind the invitation: Biden would be holding a press conference where he, along with scientists and clinicians, would unveil an initiative by President Obama to coordinate efforts to treat and cure cancer. In the tradition of the 1960s American space program that resolved to—and, in short order, did—send humans to the moon, this “cancer moonshot” aimed to rally the country’s best
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Scientists are now combining computer science and gene editing to probe the depths of the genome, hunting for new cancer-associated genes without any a priori information about them.
Our DNA is constantly changing, roiled by random, naturally occurring mutations.
Every person experiences roughly one million mutations throughout the body per second, and in a rapidly proliferating organ like the intestinal epithelium, nearly every single letter of the genome will have been mutated at least once in at least one cell by the time an individual turns sixty.
